2. Objectives
• Define pharmacoeconomics
• Differentiate various pharmacoeconomic evaluation methods
• Discuss various considerations essential to evaluating a
pharmacoeconomic analysis
• Provide examples of how pharmacoeconomics is applied in
practice and various roles for the pharmacist
3. Background - Pharmacoeconomics
Process of identifying, measuring, and comparing
costs and consequences of therapies and
determining which alternative produces best
outcomes for resources invested
4. True or False
The goal of pharmacoeconomics is to find the cheapest
alternative
6. Cost of Illness Evaluation
• Aims to identify and measure total costs attributable to a
particular disease
• Can demonstrate economic burden of disease
• Units of Measurement
o Cost – Monetary ($)
o Consequence – Not measured
7. Cost Minimization Analysis
• Compares costs for therapeutically equivalent drugs to identify
the least costly alternative
• Units of measurement
o Cost – Monetary ($)
o Consequences – assumed equivalent
8. Cost Benefit Analysis
• Primarily used to determine allocative efficiency
• Units of Measurement
o Cost – Monetary ($)
o Consequences – Monetary ($)
9. Cost Effectiveness Analysis
• Compares costs of two or more alternatives versus outcomes measured in
natural units (eg: BP, HbA1c)
• Units of measurement
o Cost – Monetary ($)
o Consequence – health outcome (eg: BP, HbA1c)
• ICER – Incremental Cost Effectiveness Ratio
ICER = ∆Cost/∆Effect
= (CTx1 – CTx2)/(ETx1 – ETx2)
10. Cost Utility Analysis
• Analysis that account for both quality and quantity of life which
are expressed as “utilities”
o Utilities represent patient preferences and quality of life associated
with disease and/or treatment
• Units of Measurement
o Cost – Monetary ($)
o Consequence – Quality adjusted life year (QALY)
• Quality adjusted life year – factor of life expectancy and utility
ICER = ∆Cost/∆Effect
= (CTx1 – CTx2)/(QALYTx1 – QALYTx2)
11. Cost Effectiveness Plane
-500
-400
-300
-200
-100
0
100
200
300
400
500
-1 -0.8 -0.6 -0.4 -0.2 0 0.2 0.4 0.6 0.8 1
More Costly, Less Effective
Quadrant II
Standard treatment dominant
More Costly, More Effective
Quadrant I
Trade off
Less Costly, Less Effective
Quadrant III
Trade off
Less Costly, More Effective
Quadrant IV
New treatment dominant
∆ Effectiveness
∆
Cost
11
12. Cost Effectiveness Plane
-$500
-$400
-$300
-$200
-$100
$0
$100
$200
$300
$400
$500
-1 -0.8 -0.6 -0.4 -0.2 0 0.2 0.4 0.6 0.8 1
More Costly, Less Effective
Quadrant II
Standard treatment dominant
More Costly, More Effective
Quadrant I
Trade off
Less Costly, Less Effective
Quadrant III
Trade off
Less Costly, More Effective
Quadrant IV
New treatment dominant
∆ Effectiveness
∆
Cost
12
13. Recap of Pharmacoeconomic Analyses
Model Type Cost Consequences Aim
Cost-minimization Monetary Assumed to be equal Efficiency
Cost-effectiveness Monetary Health Outcome Determine efficiency when
comparators are not
therapeutically equivalent
Cost-benefit Monetary Monetary Resource allocation
Cost-utility Monetary QALY Health related quality of
life gains for comparators
with similar or different
clinical outcome measures
14. Considerations for Designing or Evaluating
Pharmacoeconomic Studies
• Costs
Direct medical – e.g., medication and administration
Direct non-medical – e.g., transportation for treatment
Indirect – e.g., lost wages due to illness
Intangible – e.g., pain, suffering
• Perspective
Patient, Provider, Payer, Society
Perspective dictates what costs are considered
15. Considerations for Designing or
Evaluating Pharmacoeconomic Studies
• Discounting - value of money changes over time
A dollar is worth more today than in the future
• Sensitivity Analysis
Challenges results and tests assumptions by altering variables
• Accuracy and transparency
Clearly documented study design, assumptions, inputs
• Face Validity
Do the assumptions/input and alternatives reflect reality
16. Economic Modeling
• Analytic models used to predict economic consequences of
coverage, treatment, and access decisions
• Constructed by health plans, pharmaceutical manufacturers,
academic groups, and consultants
• These models are associated with uncertainty
18. Probabilistic Sensitivity Analysis
• Instead of changing one or more variables at a time, all
variables are simultaneously changed using a simulation
19. Applications in Practice & Roles of the
Pharmacist
• Assist in the design and implementation of research studies
• Evaluate pharmacoeconomic literature
• Apply results to clinical decision making
Individual patient care
Formulary/utilization management
Disease management
Resource allocation
20. Helpful Resources
• Navarro RP, ed. Managed Care Pharmacy Practice. 2nd edition. Jones and
Bartlett Publishers: Sudbury, MA; 2009.
• www.ispor.org
• https://icer-review.org/
• https://www.equator-network.org/wp-content/uploads/2013/04/Revised-CHEERS-
Checklist-Oct13.pdf
• Husereau D, Drummond M, Petrou S, et al. Consolidated Health Economic
Evaluation Reporting Standards (CHEERS)—Explanation and Elaboration: A
Report of the ISPOR Health Economic Evaluation Publication Guidelines Good
Reporting Practices Task Force. Value in Health. 2013; 16:231-250.
21. Conclusion
• Pharmacoeconomic evaluations consider cost compared to
consequences of treatment alternatives
• Results are used to support population-level decisions
regarding medication coverage and use
• Best-Practice principles should be used in designing
pharmacoeconomic studies to optimize transparency and
reduce bias
22. Purpose of Quality Measures
• Quality measures are a mechanism to help quantify the quality
of care provided
• Quality measures address aspects of care such as:
Utilization of evidence-based medicine
Efficiency and effectiveness
Quality of Life
Patient Satisfaction
• Useful in pay-for-performance and value-based purchasing
reimbursement programs
23. Thank you to AMCP member Pranav
Patel and Michael Pazirandeh for
updating this presentation for 2020.
24. To improve patient health by
ensuring access to
high-quality, cost-effective
medications and other therapies.
Mission & Vision
Editor's Notes
Answer is False – Goal of pharmacoeconomics is to find the therapeutic option that provides the best VALUE. Cost is just one part of the value story of a pharmaceutical product/service
Full Economic Evaluation accounts for BOTH cost and consequences
Partial Economic Evaluation accounts for EITHER cost/consequences
The analyses differ based on the unit of measurement for the outcomes. These will be discussed in detail in the upcoming slides
Can be used to quantify disease burden for various stakeholders. For example, it can quantify cost of opioid abuse/smoking for legal settlements.
Advantages: Ease-of-use
Disadvantages: Few alternatives are known to have the “same” outcomes
Advantages: Comprehensive, Allows comparison of different interventions which may not have similar outcomes for measurement. Converting these outcomes into $$ can help level the field to compare the interventions
Disadvantages: Complex; the patient experience is hard to convert into $$
Advantages: Most commonly used form of analysis
Disadvantages: Unable to compare different programs or therapeutic classes, Unable to judge “true” worth.
ICER - Incremental cost to achieve a one unit increase in outcome
Advantages: Able to evaluate years of life as well as quality of life
Disadvantages: Concerns with validity
QALY - e.g., 4 years at 25% QoL = 1 year at 100% QoL
Utility weights are quantified using questionnaires like SF36 or EURQoL. These may be general or disease-specific.
Point to note – this pharmacoeconomic evaluation adopts a drug in Quadrant 1 – which is MORE costly and MORE effective. This shows that the aim of pharmacoeconomics is not to find the CHEAPEST alternative.
Direct Health Care Costs: Drug, services, health care professionals time, health care facilities
Direct Non-Health Care Costs: Private transportation, patient and family time, special diet
Indirect Costs: Productivity losses related to illness, Productivity losses related to death, Different than the “accounting” concept
Intangible Costs: Costs associated with pain and suffering
Perspective – drives costs and consequences considered
Discounting Costs & Outcomes: Allowance for the development of the differential effects of the options
Sensitivity Analysis: Changing the value of variables to account for uncertainty; identifies what factors, which if changed, impact conclusions
Sensitivity analysis – these analyses or models assume multiple items. In real world, these assumptions may not be true. Sensitivity analysis is a technique where these assumptions are varied to see how the ICERs change with it. Sensitivity analyses allows the researchers to test how robust their findings are if they were to vary the assumptions.
Economic models are utilized to get ICERs in many cases. Once these models are created, values (from clinical trials and other evidence) are fed into these models.
These values may not represent every single patient/case and are associated with a lot of uncertainty. A robust pharmacoeconomic analysis tries to address the issues of uncertainty using sensitivity analysis.
There are 2 kinds of sensitivity analysis
One way
Probabilistic
In one way sensitivity analysis, the input values are changed one at a time while keeping others a constant. The results of such a sensitivity analysis is presented as a tornado diagram. In the tornado diagram above, the dotted line represents the base case ICER value. Base case ICER represents the ICER value obtained based on the values fed into the model. In this analysis, the model included
Cost for HIV tests
Start up costs
Cost/counseling session …….and the other variables mentioned on the y-axis
The numbers at the end of each bar represents the range over which each of the variables was changed. We can see that ICER changes are sensitive to changes in HIV incidence, discount rate, and intervention effectiveness
Burgos JL, Gaebler JA, Strathdee SA, Lozada R, Staines H, et al. (2010) Cost-Effectiveness of an Intervention to Reduce HIV/STI Incidence and Promote Condom Use among Female Sex Workers in the Mexico–US Border Region. PLOS ONE 5(6): e11413. https://doi.org/10.1371/journal.pone.0011413
One-way sensitivity analysis is not how things happen in the real-world. We do not change one parameter at a time in reality. All variables are dynamic in nature. To address this drawback of one-way sensitivity analysis, we have probabilistic sensitivity analysis,
Here, instead of changing one or more variables at a time, we change all the variables simultaneously using a simulation.
The results of this sensitivity analysis can be shown in the following manner. The base case analysis can be found in the second quadrant. Each blue dot represents an ICER value for one set of simulation. In this example, there were 10,000 simulations that gave us the above scatter plot.
Jordan B. King, Rashmee U. Shah, Adam P. Bress, Richard E. Nelson, Brandon K. Bellows. Cost-Effectiveness of Sacubitril-Valsartan Combination Therapy Compared With Enalapril for the Treatment of Heart Failure With Reduced Ejection Fraction. J Am Coll Cardiol HF. 2016 May, 4 (5) 392-402.
For students – there are multiple opportunities within pharma (market access, HEOR) and managed care (analytics and outcomes, P&T, HEOR). Typical career path for those interested in pharmacoeconomics is a fellowship/residency. Additionally, students could pursue MPH/MS/PhD to gain quantitative skills for these roles.